The compound, originally patented by VCU under the name 5-HMF, was developed by a team from the VCU Institute for Structural Biology and Drug Discovery, an interdisciplinary research center spanning the School of Medicine and the School of Pharmacy. The team included Martin Safo, Ph.D., associate professor ofmedicinal chemistry; Richmond Danso-Danquah, Ph.D., assistant professor of medicinal chemistry; and Donald Abraham, Ph.D., the Alfred and Frances P. Burger Emeritus Professor of Medicinal Chemistry and Biological Chemistry and emeritus director of the institute.

“TRND researchers have worked collaboratively with AesRx to provide preclinical drug development expertise and clinical and regulatory resources necessary to move Aes-103 into human clinical trials,” said John McKew, Ph.D., acting director of NCATS Division of Preclinical Innovation and director of TRND. “Through TRND, NCATS supports innovative methods and collaborative approaches to accelerate the discovery process while developing promising treatments for the patients who need them.”

Sickle cell disease is a recessive disorder of the hemoglobin that can lead to a wide range of serious, sometimes life-threatening, conditions, including chronic hemolytic anemia, chronic pain and acute painful crisis, stroke, acute chest syndrome and cumulative damage to tissues and organs. More than 100,000 people in the United States are afflicted with sickle cell disease.

“Francis Collins, M.D., Ph.D., the director of the National Institutes of Health, has held this project up as a prime example of translational medicine with academic medical centers working with startup companies in the research and development of life-changing drug therapies,” said Francis Macrina, Ph.D., VCU vice president for research.